Clinical Trial

Safety and Efficacy of AAV9. hMCOLN1co For Patients With Mucolipidosis Type IV

Name: Safety and Efficacy of AAV9. hMCOLN1co For Patients With Mucolipidosis Type IV — clinical trial record (NCT07398872)
Creator: DataLookout
License: https://creativecommons.org/licenses/by/4.0/

Study acronym: AAV9-hMCOLN1

Enrolling by Invitation Phase 1

View on ClinicalTrials.gov →

Summary

Safety and Efficacy of AAV9.hMCOLN1co for patients with Mucolipidosis Type IV(MLIV): A Single-Center, Interventional, Open-Label, Single-Arm Clinical Study. The goal of this clinical trial is to evaluate whether a gene therapy can safely treat children with MLIV.

Trial Details

NCT Number	NCT07398872
Lead Sponsor	The Children's Hospital of Zhejiang University School of Medicine
Conditions	Mucolipidosis Type IV
Enrollment	1 participants
Start Date	2026-01-13
Primary Completion	2027-01-20 (estimated)
Study Completion	2031-01-20 (estimated)
Updated on ClinicalTrials.gov	2026-02-10